FDA Grants Revalesio Orphan Drug Status for ALS Investigational Drug RNS60

TACOMA, Wash. – September 20, 2017 – Revalesio announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for the investigational drug RNS60 for the treatment of amyotrophic lateral sclerosis (ALS).

The Orphan Drug Designation program provides incentives for pharmaceutical companies to develop products to treat rare diseases and disorders that affect fewer than 200,000 people in the U.S.[1] The program has driven market approval of more than 60 drugs for rare diseases since 1983.

“With orphan status, we have increased access to FDA to help facilitate RNS60’s drug development plan and, hopefully, bring RNS60 to patients who are suffering from ALS,” said Greg Archambeau, President of Revalesio.

ALS is a fatal degenerative disorder of large motor neurons of the cerebral cortex, brain stem, and spinal cord. The disease, which is also known as Lou Gehrig’s disease, leads to progressive paralysis of voluntary muscles. ALS affects approximately 15,000 patients in the United States. Only two drugs have been approved for ALS since 1995, and no available treatment has shown to halt, reverse, or prevent the disease.

RNS60 is an investigational drug approved for clinical evaluation in the U.S. and in Europe. The investigational drug has shown profound anti-inflammatory and neuroprotective effects in preclinical disease models of ALS and other neurodegenerative diseases. The compound has been exceptionally well tolerated in clinical studies to date.

“Our treatment impacts the underlying energetic deficits in cells, rather than a single molecular target,” said Archambeau. “Having personally seen the devastating effects of ALS in a Revalesio colleague, we are motivated to find treatments that improve the health and quality of life for patients with ALS.”

RNS60 is currently being evaluated in an exploratory open label study in ALS patients at Massachusetts General Hospital (MGH)[2]. A larger, 142-patient placebo-controlled, double-blind study is evaluating the effects of RNS60 on biomarkers of ALS and their correlation with disease progression at 20 clinical centers in Italy and at MGH[3]. This study is funded in part by a $1 million grant from the ALS Association (supported by the Ice Bucket Challenge) in partnership with the Northeast ALS Consortium and ALS Finding a Cure[4].

Revalesio has retained Torreya Partners to explore business development activities, including licensing and partnering for RNS60. Revalesio and Torreya will be available for discussions at BioPharm America in Boston on September 26th and 27th.

 

About Revalesio Corporation

Revalesio is a pioneering clinical stage biomedical company dedicated to improving the lives of patients with neurodegenerative diseases through developing safe and effective new treatments. Founded in 2007 and based in Tacoma, Washington, Revalesio has partnered with leaders in biomedical research around the world to develop RNS60. Revalesio has an extensive patent portfolio on a novel class of anti-inflammatory and neuroprotective products. For more information, visit revalesio.com.

About Torreya Partners

Torreya Partners is a global investment banking boutique serving companies in the life sciences industry. Since its inception in 2007, the firm has closed more than $100 billion worth of deals for biotechnology including branded pharmaceutical, generic pharmaceutical, and life sciences companies. Our partners are senior industry bankers and executives, with deep experience, knowledge, and networks. We are a partner of choice for companies seeking discreet, conflict-free and knowledgeable advice on M&A, pharmaceutical asset sale, capital markets, and licensing transactions.  Torreya is differentiated from most other life sciences advisory practices by the breadth of its global presence. Over half of the transactions on which we advise are cross-border. Torreya has offices in London, Mumbai and New York, and affiliate offices on six continents.

[1] https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm

[2] https://clinicaltrials.partners.org/study/9709

[3] https://alsadotorg.wordpress.com/2017/09/19/clinical-trial-als-association-supported-phase-ii-rns60-trial-is-now-enrolling/

[4] http://www.alsa.org/news/media/press-releases/rns60-treatment-120216.html